NEW YORK (Reuters Health) - Gene silencing with RNA interference could represent a useful therapy for Huntington’s disease, findings from an animal study suggest.
In the study, silencing of the huntingtin (htt) gene improved motor function as well as the neuropathologic abnormalities seen in affected mice.
“RNA interference has emerged as a potential therapeutic tool for treating dominant disease by directly reducing disease gene expression,” senior author Dr. Beverly L. Davidson, from the University of Iowa in Iowa City, and colleagues note.
The researchers created short hairpin RNAs directed against mutant human htt. When tested in vitro and in a murine model of Huntington’s disease, this approach reduced htt mRNA and protein expression. Most importantly, this RNA interference improved the behavioral abnormalities seen in the affected animals.
The new findings appear in the April 4th Early Edition of the Proceedings of the National Academy of Sciences.
“Our data suggest the feasibility of treating Huntington’s disease by directly reducing mutant htt gene expression by using RNA interference and support its general applicability to treating other dominant neurodegenerative disorders,” the authors conclude.
Source: Proc Natl Acad Sci USA 2005. [ Google search on this article ]
MeSH Headings:Animal Diseases: Biological Sciences: Biology: Disease Models, Animal: Gene Expression Regulation: Genetics: Genetics, Biochemical: Molecular Biology: Gene Silencing: Biological Sciences: DiseasesCopyright © 2002 Reuters Limited. All rights reserved. Republication or redistribution of Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Reuters. Reuters shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon. Reuters and the Reuters sphere logo are registered trademarks and trademarks of the Reuters group of companies around the world.
